Drug intelligence / Profile preview

osilodrostat

Development stage
Approved
Lead developer
Novartis
Modality
Small Molecules
Administration
Oral
01

Overview

Osilodrostat is an orally active, nonsteroidal small molecule inhibitor of cortisol synthesis used to treat adults with Cushing's disease who cannot undergo pituitary surgery or have persistent disease after surgery. It acts primarily by inhibiting 11β-hydroxylase (CYP11B1), the enzyme responsible for the final step in cortisol biosynthesis in the adrenal gland, and to a lesser extent aldosterone synthase (CYP11B2). By blocking these enzymes, osilodrostat reduces excessive cortisol production characteristic of Cushing's disease. Developed by Novartis, it offers a treatment option for patients not eligible for or not cured by pituitary surgery. Osilodrostat has demonstrated rapid and sustained control of hypercortisolism and improvement in related symptoms such as hypertension and metabolic disturbances[1][2][6][7].

Brand names
Isturisa
Other names
osilodrostatLCI-699LCI699LCI 699
02

Targets

CYP11A1 (Cholesterol side chain cleavage enzyme)CYP11B2 (Cytochrome P450 11B2)CYP11B1 (Cytochrome P450 11B1)

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